Stockwinners Market Radar for September 06, 2021 - Earnings, Upgrades downgrades, option trades, Best Stock Advisory Service

16:18 EDT TBA of EPO dismisses Forward's appeal of EP2801355 patent decision - Forward Pharma (FWP) announced that the Technical Board of Appeal of the European Patent Office has dismissed Forward's appeal of the previous decision of the EPO Opposition Division to revoke the EP2801355 patent following the oral hearing. The TBA made its decision after considering Forward's appeal against the decision of the Opposition Division and third-party submissions from several opponents. The TBA will issue detailed reasons for the decision in written form in due course, and following receipt and review of these, Forward will announce future plans for the company. Such plan may involve a petition for review at the Enlarged Board of Appeal of the EPO in an effort to overturn the unfavorable outcome, but the likelihood of a petition for review being successful is low. The denial of a petition for review would end the Opposition Proceeding in favor of the opponents. For all practical purposes, such denial or the absence of a petition for review would represent an unsuccessful outcome of the Opposition Proceeding, resulting in no royalties being due to the company from Biogen (BIIB) based on Biogen's future net sales outside the United States, as defined in the Settlement and License Agreement by and among Forward, subsidiaries of Biogen and certain other parties thereto.

16:06 EDT FDA places clinical hold on BioMarin '307 Phearless Phase 1/2 study - BioMarin Pharmaceutical announced that the U.S Food and Drug Administration placed a clinical hold on the BMN 307 Phearless Phase 1/2 study. The Phearless study is evaluating BMN 307, an investigational AAV5-phenylalanine hydroxylase gene therapy, in adults with phenylketonuria. The FDA's clinical hold was based on interim safety findings from a pre-clinical, non-GLP pharmacology study. The company carried out this pre-clinical study to understand the durability of BMN 307 activity in mice bearing two germline mutations, which may predispose the mice to the development of malignancy. One mutation eliminated the PAH gene that's missing in PKU and the second rendered the animals immunodeficient. Of 63 animals treated, six of seven animals administered BMN 307 at the highest dose group had tumors on liver necropsy 52 weeks after dosing with evidence for integration of portions of AAV vector into the genome. No lesions were observed in any mice at 24 weeks. Five of these animals had adenomas and one had a hepatocellular carcinoma. The translatability of these findings to humans is uncertain and under further investigation. To date, the company has only dosed humans in the Phearless Phase 1/2 clinical study with lower doses of either 2e13 vg/kg or 6e13 vg/kg. Due in part to the risk previously identified by historical rodent studies, the liver health of Phearless study participants is regularly monitored. The company will work with the Data Review Board and Principal Investigators to further evaluate the study participants who have been dosed and will continue to monitor them over the long-term. The clinical significance of these pre-clinical rodent findings has not been established and cancers due to AAV integration have not been observed in larger animals or humans. BioMarin is pausing further enrollment into this global Phase 1/2 study until the investigation of these findings is completed. The company is working with the FDA and other health authorities and will communicate next steps for the program when available. "More than 3,000 patients have been treated with gene therapy, and there are no reports of cancers emerging as a consequence. Acknowledging the complexity of the issue as highlighted in this week's FDA discussion, integrational mutagenesis and resultant cancer formation has been observed in mice using other AAV vectors," said Hank Fuchs, President, Worldwide Research and Development at BioMarin. "Therefore, we plan to investigate these findings. For patients who have already received lower doses of these vectors, we will continue to carefully evaluate and monitor their health. We are committed to understand and mitigate any risk of cancer causation."